Regulatory Collaborations Aim to Speed Access to New Vaccines and Drugs

The need for treatments to combat the spread of COVID-19 is promoting greater cooperation among drug regulatory authorities around the world. FDA officials are communicating more frequently with their counterparts in Europe, Canada, Japan, and other nations through established programs and agreements. In addition, the International Coalition of Medicines Regulatory Authorities (ICMRA) has expanded its program to facilitate regular discussions among some 30 governmental and international agencies. These efforts seek common approaches to clinical research and biopharmaceutical production needed to wipe out the coronavirus pandemic globally.

The long-standing collaboration between FDA and the European Commission (EC) and the European Medicines Agency (EMA) has shifted to promote regulatory cooperation to combat COVID-19. A high-level FDA-EMA virtual meeting in mid-June reviewed ongoing efforts to develop new therapeutics and vaccines, as well as the use of real-world data to inform regulatory decisions. A main focus was the challenges related to production and rapid scale-up of potential vaccines and therapeutics.

FDA and EU efforts to address COVID issues also is advancing through the work of some 30 FDA-EMA technical expert groups that regularly address topics such as vaccine development, pharmacovigilance, blood products, and drug shortages. Now these panels are examining timely topics like interpretation of data supporting emergency use authorization of experimental treatments, expanded use of real-world data to better understand the natural history of the coronavirus, and the performance of diagnostics. These discussions often include regulatory authorities from Japan, Canada, Australia, and other established agencies.

In addition, FDA and EMA officials have extended the discussion of these and other topics to a broader group of regulatory authorities through ICMRA. Since March, experts representing 25 agencies in 17 countries have participated in regular virtual meetings to gain understanding and convergence of requirements on clinical trials and data standards for COVID-19 vaccines and therapeutics, plus the use of real-world evidence and observational studies to complement evidence from clinical trials. ICMRA members have discussed what preclinical data is needed to support proceeding to first-in-human clinical trials and the importance of risk mitigation strategies to better protect research participants. These experts further discussed guiding principles for COVID clinical trials, including the use of master protocols, ethical questions related to human challenge trials, post-approval requirements for vaccines, and the need for regulatory flexibility during the pandemic, at a meeting in June, which was moderated by Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), but now on detail to the White House Operation Warp Speed initiative.

A main goal of such interactions is for regulators in different regions to understand and accept the study designs and clinical endpoints of early vaccine candidates, pointed out Peter Marks, director of the Center for Biologics Evaluation and Research (CBER), at the annual meeting of the Biotechnology Innovation Organization (BIO) in June. He emphasized that COVID vaccine development “is really a global issue,” and that it’s important for authorities around the world to gain an understanding about “what success will look like” in bringing safe and effective preventives to market.

Because these vaccines may be widely used after only months—instead of years—of testing, Marks acknowledged that clinical trials will need to be sufficiently large and widespread and closely monitor early vaccine subjects to evaluate safety effectively.

And multiple vaccines may gain approval, as some populations may benefit from one preventive more than from another. A common understanding of the need to test any emerging vaccine on a sufficient number to establish a strong safety dataset will help avoid later debate among authorities over which assays to consider or the size of studies needed, Marks noted.

Equitable access

Even with a high degree of international collaboration on advancing vaccine development and production, a certain degree of “vaccine nationalism” will affect the distribution of any successful preventive. Research and regulatory authorities recognize that biopharma companies and government health agencies will be under pressure to take care of their home populations first, but they also expect industry to ramp up production and form partnerships with other organizations to fully supply broader regions of the world.

Huge investments by private firms and government agencies are being made to retrofit existing facilities and to secure supplies of key raw materials to be able to produce billions of dosages. At the same time, researchers seek strategies for producing more stable products that can be stored longer and shipped more easily, as well as formulations that can provide needed protection with a smaller dose.

To ensure early distribution of COVID vaccines in all regions, the World Health Organization (WHO), the Global Alliance Vaccine Initiative and the Coalition for Epidemic Preparedness and Innovation (CEPI) have formed a vaccine purchasing group that seeks $18 billion for a COVID-19 advanced market commitment.

At the same time, the research community is looking to provide strong safety data in order to overcome a visible degree of public skepticism about the value of being vaccinated. “If 30% or 40% of the population will not take this vaccine,” Marks lamented at BIO, it may not be possible to achieve desired herd immunity on a broad basis.

The larger concern for Marks and others, though, is that success in combatting COVID won’t come fast enough to avoid an even more lethal second wave at the end of the year.

Jill Wechsler is Applied Clinical Trials' Washington Editor